When Rebekah and Evan Lockard’s daughter, Naomi, was diagnosed with a devastating ultra-rare genetic disease, they didn’t know where to turn. Then they found Terry Pirovolakis, an IT professional who had made a gene therapy for his son with the same disease. But the process of getting Naomi treated has been an uphill battle, full of financial and logistical obstacles. The Lockard’s story flips the question we’ve been asking all season on its head. Instead of wondering, "if we could do something, should we," we're now asking, "if we can do something that helps patients, should we do it at any cost?" And this question isn’t for scientists or researchers, it’s for the rest of us. Credits Host: <a href= "https://www.sciencehistory.org/profile/alexis-j-pedrick">Alexis Pedrick</a> Executive Producer: <a href= "https://www.sciencehistory.org/profile/mariel-carr">Mariel Carr</a> Producer: <a href= "https://www.sciencehistory.org/profile/rigoberto-hernandez">Rigoberto Hernandez</a> Associate Producer: <a href= "https://www.sciencehistory.org/profile/sarah-kaplan/">Sarah Kaplan</a> Audio Engineer: <a href="https://samiabouzid.com/" target= "_blank" rel="noreferrer noopener">Samia Bouzid</a> Music by <a href="https://www.sessions.blue/" target= "_blank" rel="noreferrer noopener">Blue Dot Sessions</a> Resource List Mast, Jason. "<a href= "https://www.statnews.com/2024/12/09/pirovolakis-gene-therapy-rare-disease-spg50-lockard/" target="_blank" rel="noreferrer noopener">A dad built a gene therapy for his son. Can he save other kids, too?</a>" STAT News. <a href="https://www.elpidatx.com/about" target="_blank" rel= "noreferrer noopener">Elpida Therapeutics</a>. "<a href= "https://www.jax.org/news-and-insights/2022/August/battling-spg50-and-changing-the-world" target="_blank" rel="noreferrer noopener">Battling SPG50 and changing the world</a>." August 17, 2022. The Jackson Laboratory. Brent, Jonathan R. and Deng, Han-Xiang. "<a href= "https://pmc.ncbi.nlm.nih.gov/articles/PMC10178831" target="_blank" rel="noreferrer noopener">Paving a way to treat spastic paraplegia 50</a>." "<a href="https://www.nature.com/articles/s41591-024-03078-4" target="_blank" rel="noreferrer noopener">AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient</a>." June 28, 2004. "<a href= "https://www.cbsnews.com/colorado/news/colorado-family-funding-awareness-rare-neurological-disorder-spastic-paraplegia-50-spg-50/" target="_blank" rel="noreferrer noopener">Colorado family pushes for more funding, awareness around rare neurological disorder</a>." August 12, 2024. CBS News Colorado. <a href="https://www.naomiscorner.org/about" target="_blank" rel="noreferrer noopener">Naomi's Corner</a>.

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